GPH101, now called nulabeglogene autogedtemcel
(nula-cel), designed to directly correct the genetic mutation that
causes sickle cell disease
Initial proof-of-concept data from Phase 1/2
CEDAR trial anticipated in mid-2023
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company harnessing the power of
high-efficiency precision gene repair to develop therapies with the
potential to cure serious diseases, today announced that the first
patient has been dosed with GPH101, now called nulabeglogene
autogedtemcel (nula-cel), in the company’s Phase 1/2 CEDAR trial in
people with sickle cell disease (SCD). Nula-cel is an
investigational gene editing therapy designed to directly correct
the genetic mutation that causes SCD and definitively cure the
disease.
“For decades, the goal of gene editing has been to precisely
correct genetic mutations that cause disease. Today, we took an
important step toward achieving that goal by dosing our first
patient with nula-cel, the first investigational therapy designed
to correct a mutated gene to normal. This first use of
high-efficiency precision DNA repair to correct a genetic mutation
is an important milestone not only for our company but also for the
gene editing field and, hopefully, for the sickle cell community,”
said Josh Lehrer, M.D., M.Phil., chief executive officer of
Graphite Bio.
“We continue to make tremendous progress with the development of
nula-cel, which in preclinical studies successfully corrected the
sickle cell disease mutation, directly reducing sickle hemoglobin
and restoring healthy adult hemoglobin to potentially curative
levels,” Lehrer continued. “We believe nula-cel could be a
definitive cure for sickle cell disease, with the potential to
address all complications associated with this life-threatening
disease. We look forward to reporting initial proof-of-concept data
from the CEDAR trial in mid-2023.”
The CEDAR trial is a Phase 1/2 open-label, single-dose clinical
trial evaluating the safety, preliminary efficacy and
pharmacodynamics of nula-cel in approximately 15 patients with
severe SCD. The trial is currently enrolling patients at multiple
sites in the United States.
About nula-cel
Nula-cel, formerly known as GPH101, is an investigational
next-generation gene-editing autologous hematopoietic stem cell
(HSC) therapy designed to directly correct the genetic mutation
that causes sickle cell disease (SCD). A serious, life-threatening
inherited blood disorder, SCD affects approximately 100,000 people
in the United States and millions of people around the world,
making it the most prevalent monogenic disease worldwide. Nula-cel
is the first investigational therapy to use a highly differentiated
gene correction approach that seeks to efficiently and precisely
correct the mutation in the beta-globin gene to decrease sickle
hemoglobin (HbS) production and restore adult hemoglobin (HbA)
expression, thereby potentially curing SCD. The U.S. Food and Drug
Administration (FDA) granted Fast Track and Orphan Drug
designations to nula-cel for the treatment of SCD.
Graphite Bio is evaluating nula-cel in the CEDAR trial, an
open-label, multi-center Phase 1/2 clinical trial designed to
assess safety, engraftment success, gene correction rates, total
hemoglobin, as well as other clinical and exploratory endpoints and
pharmacodynamics in patients with severe SCD.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing
company driven to discover and develop cures for a wide range of
serious and life-threatening diseases. The company is pioneering a
precision gene editing approach that has the potential to transform
human health by achieving one of medicine’s most elusive goals: to
precisely “find & replace” any gene in the genome. Graphite
Bio’s UltraHDR™ gene editing platform takes CRISPR beyond cutting
and harnesses the power of high-efficiency precision DNA repair,
also known as homology directed repair (HDR), to precisely correct
genetic mutations, replace entire disease-causing genes with
functional genes or insert new genes into predetermined, safe
locations. The company was co-founded by academic pioneers in the
fields of gene editing and gene therapy, including Maria Grazia
Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.
Learn more about Graphite Bio by visiting www.graphitebio.com
and following the company on LinkedIn and Twitter.
Forward-Looking Statements
Statements we make in this press release may include statements
which are not historical facts and are considered forward-looking
statements within the meaning of Section 27A of the Securities Act
of 1933, as amended (the “Securities Act”), and Section 21E of the
Securities Exchange Act of 1934, as amended (the “Exchange Act”).
These statements may be identified by words such as “aims,”
“anticipates,” “believes,” “could,” “estimates,” “expects,”
“forecasts,” “goal,” “intends,” “may,” “plans,” “possible,”
“potential,” “seeks,” “will,” and variations of these words or
similar expressions that are intended to identify forward-looking
statements. Any such statements in this press release that are not
statements of historical fact, including statements regarding our
nula-cel (formerly GPH101) product candidate, its clinical and
therapeutic potential, our plans to advance nula-cel in our Phase
1/2 CEDAR trial and to report initial proof-of-concept data, and
the timing of these events, may be deemed to be forward-looking
statements. We intend these forward-looking statements to be
covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act and
Section 21E of the Exchange Act and are making this statement for
purposes of complying with those safe harbor provisions.
Any forward-looking statements in this press release are based
on Graphite Bio’s current views about our plans, intentions,
expectations, strategies and prospects only as of the date of this
release and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements,
including the risk that we may encounter regulatory hurdles or
delays, for example, in patient enrollment and dosing, and in the
progress, conduct and completion of our Phase 1/2 CEDAR trial and
our other planned clinical trials. These risks concerning Graphite
Bio’s programs and operations are described in additional detail in
its periodic filings with the SEC, including its most recently
filed periodic report, and subsequent filings thereafter. Graphite
Bio explicitly disclaims any obligation to update any
forward-looking statements except to the extent required by
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20220811005244/en/
Company Contact: Stephanie Yao VP, Communications and
Investor Relations 443-739-1423 syao@graphitebio.com
Investors: Stephanie Ascher Stern IR, Inc. 212-362-1200
ir@graphitebio.com
Media: Sheryl Seapy Real Chemistry 949-903-4750
media@graphitebio.com
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